{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T16:24:28Z","timestamp":1793982268668,"version":"4.8.0"},"reference-count":34,"publisher":"American Society of Hematology","issue":"4","license":[{"start":{"date-parts":[[2024,11,1]],"date-time":"2024-11-01T00:00:00Z","timestamp":1730419200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,1,23]]},"abstract":"<jats:sec>\n                  <jats:title>Abstract<\/jats:title>\n                  <jats:p>Cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA) are rare autoimmune hemolytic anemias characterized by red blood cell destruction, largely attributable to complement activation resulting in intravascular and extravascular hemolysis. Pegcetacoplan is a subcutaneously administered C3-targeted therapy, which may be suitable for treating CAD and wAIHA. In this open-label phase 2 study, analyses were conducted in 2 cohorts, 1 for patients with CAD and the other for those with wAIHA. In each cohort, patients were randomly assigned to receive pegcetacoplan 270 mg\/d or 360 mg\/d for up to 48 weeks. Safety end points included the incidence and severity of treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESI). Efficacy end points included change from baseline in hemoglobin (Hb), lactate dehydrogenase, absolute reticulocyte count, haptoglobin, indirect bilirubin, and functional assessment of chronic illness therapy (FACIT)-fatigue scale. Thirteen of 13 (100%) and 10 of 11 (91%) patients with CAD and wAIHA, respectively, experienced at least 1 TEAE. Ten patients had at least 1 serious AE; none were considered related to pegcetacoplan. The only treatment-related AESIs were injection site reactions. Pegcetacoplan increased Hb levels, reduced hemolysis, and increased FACIT-fatigue scale scores in the first weeks; at week 48 the median (interquartile range) change from baseline Hb for the CAD and wAIHA total groups was 2.4 (0.90-3.00) and 1.7 g\/dL (\u22121.40 to 2.90), respectively, and improvements in hemolysis and FACIT-fatigue scale scores were maintained. This study demonstrated that pegcetacoplan is generally well tolerated and suggests it can be effective for patients with CAD and wAIHA. This trial was registered at www.ClinicalTrials.gov as #NCT03226678.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1182\/blood.2023022549","type":"journal-article","created":{"date-parts":[[2024,11,1]],"date-time":"2024-11-01T21:23:03Z","timestamp":1730496183000},"page":"397-408","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":24,"title":["Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemia"],"prefix":"10.1182","volume":"145","author":[{"given":"Eloy","family":"Roman","sequence":"first","affiliation":[{"name":"1Hematology-Oncology, Lakes Research, Miami Lakes, FL"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0857-8379","authenticated-orcid":false,"given":"Bruno","family":"Fattizzo","sequence":"additional","affiliation":[{"name":"2Hematology Unit, Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Milan, Italy"},{"name":"3Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Merrill","family":"Shum","sequence":"additional","affiliation":[{"name":"4Cancer and Blood Specialty Clinic, The Oncology Institute of Hope and Innovation, Whittier, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wahid","family":"Hanna","sequence":"additional","affiliation":[{"name":"5Hematology\/Oncology, University of Tennessee Medical Center, Knoxville, TN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8885-4718","authenticated-orcid":false,"given":"Steven R.","family":"Lentz","sequence":"additional","affiliation":[{"name":"6Department of Internal Medicine, University of Iowa, Iowa City, IA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sergio Schusterschitz S.","family":"Araujo","sequence":"additional","affiliation":[{"name":"7Centro de Pesquisas Cl\u00ednica, Hospital das Cl\u00ednicas da Universidade Federal de Minas Gerais, Belo Horizonte, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohammed","family":"Al-Adhami","sequence":"additional","affiliation":[{"name":"8Biostatistcs, Apellis Pharmaceuticals, Waltham, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico V.","family":"Grossi","sequence":"additional","affiliation":[{"name":"9Clinical Research, Apellis Pharmaceuticals, Waltham, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3853-5196","authenticated-orcid":false,"given":"Morie A.","family":"Gertz","sequence":"additional","affiliation":[{"name":"10Division of Hematology, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"key":"2025012316202929300_bib1","doi-asserted-by":"crossref","first-page":"2040620719873321","DOI":"10.1177\/2040620719873321","article-title":"Novel insights into the treatment of complement-mediated hemolytic anemias","volume":"10","author":"Berentsen","year":"2019","journal-title":"Ther Adv Hematol"},{"issue":"12","key":"2025012316202929300_bib2","first-page":"3859","article-title":"New insights in autoimmune hemolytic anemia: from pathogenesis to therapy stage 1","volume":"9","author":"Barcellini","year":"2020","journal-title":"J\u00a0Clin Med"},{"issue":"3","key":"2025012316202929300_bib3","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1111\/bjh.14478","article-title":"The diagnosis and management of primary autoimmune haemolytic anaemia","volume":"176","author":"Hill","year":"2017","journal-title":"Br J Haematol"},{"key":"2025012316202929300_bib4","doi-asserted-by":"crossref","first-page":"100648","DOI":"10.1016\/j.blre.2019.100648","article-title":"Diagnosis and treatment of autoimmune hemolytic anemia in adults: recommendations from the First International Consensus Meeting","volume":"41","author":"J\u00e4ger","year":"2020","journal-title":"Blood Rev"},{"issue":"8","key":"2025012316202929300_bib5","doi-asserted-by":"crossref","first-page":"2925","DOI":"10.1182\/blood-2003-10-3597","article-title":"Rituximab for primary chronic cold agglutinin disease: a prospective study of 37 courses of therapy in 27 patients","volume":"103","author":"Berentsen","year":"2004","journal-title":"Blood"},{"issue":"4","key":"2025012316202929300_bib6","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1182\/blood-2017-04-778175","article-title":"Bendamustine plus rituximab for chronic cold agglutinin disease: results of a Nordic prospective multicenter trial","volume":"130","author":"Berentsen","year":"2017","journal-title":"Blood"},{"key":"2025012316202929300_bib7","volume-title":"Enjaymo. 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